New Therapy Alleviates the Symptoms of Cystic Fibrosis

The new model is based on the understanding that people with CF lack the cystic fibrosis transmembrane conductance regulator (CFTR), a cell "door” that is responsible for glutathione (GSH) transport. The researchers hypothesized that mutations of the CFTR, which create abnormal GSH transport, lead to aberrations of GSH levels in both the intracellular as well as the extracellular milieu. These alterations in normal cellular GSH levels affect the redox state of the cell, thereby affecting the intracellular stress protein, metallothionein.

This disruption will naturally prevent the delivery of zinc as a cofactor for various enzymatic processes, and cause alterations in both humoral and cell-mediated immunity. Moreover, the symptom of thick sticky mucus in CF patients might be explained through the understanding that oversulfation of mucus is a direct result of elevated cellular GSH and cysteine. The issues of hyperinflammation, altered pH, and the imbalance of fatty acids that are typical in CF can also be linked to disruptions in GSH homeostasis. The researchers propose a new therapy that makes use of a similar pore, the multidrug- resistant protein (MDR), to remove from the cell what normally would go through the CFTR. This is achieved using a class of compounds known as isothiocyanates. The new research was published in the January 2007 issue of The Journal of Medical Hypotheses.

"We are very excited about this therapy. For the first time ever, there may be one pill to take per day, which will correct the imbalances in CF cells. If these imbalances can be corrected with this therapy, it might be possible to eliminate the need for the plethora of medical procedures and hospitalizations these patients have to undergo, beginning at a very early age,” said lead author Melanie Childers.

Cystic Fibrosis affects the respiratory and digestive systems by the production of thick, sticky mucus that clogs the lungs and causes respiratory infections, eventually leading to an early death (median survival age is 38.6 years). One in 30 people are carriers of the CF gene and one in 2500 babies are born with CF.



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